Injecting medicines into amniotic fluid may be a viable strategy for treating SMA before birth, according to a new study in ...

Making a situation accessible is more about mindset than things, says columnist. That's why attitude is important.

SMA gene therapy GC101 was found to rescue disease manifestations in mice and to be safe in non-human primates.

Blood creatinine-to-cystatin-c ratio, an indicator of muscle mass and strength, may be a treatment response biomarker of SMA, ...

Blood creatinine-to-cystatin-c ratio, an emerging indicator of muscle mass and strength, may be a promising biomarker of therapeutic response in adults with spinal muscular atrophy (SMA), a study in ...

Training with a wearable robotic device called HAL, for hybrid assistive limb, improved walking ability for people with spinal muscular atrophy (SMA) who were also receiving treatment with Spinraza ...

With a fresh cup of coffee balanced on my knee, I quickly scanned my church’s cafe area for a place to land where I could have a supportive tabletop. The only vacant spot at a table was next to a ...

In May 1997, my husband, Randy, and I awaited the arrival of our third baby. The ultrasound indicated we were having a boy. We named him Jeffrey. Years before, I’d taught at Brockman School, a ...